Extracellular vesicles as therapeutic agents for retinal ganglion cell degeneration: current challenges and future prospects.

BACKGROUND: Retinal ganglion cells (RGCs) play a crucial role in the transmission of visual information and are particularly susceptible to degeneration in optic neuropathies such as glaucoma. Current therapeutic strategies primarily focus on lowering intraocular pressure (IOP), the only clinically proven modifiable risk factor, but often fail to completely prevent progressive RGC loss.

OBJECTIVE: This review aims to explore the potential of extracellular vesicles (EVs) as a complementary therapeutic approach for RGC degeneration in glaucoma, highlighting their advantages and the challenges associated with their application.

METHODS: A comprehensive literature review was conducted to assess the properties of EVs, their therapeutic cargo capabilities, and their implications for RGC neuroprotection.

RESULTS: EVs are naturally occurring nano-sized particles that exhibit biocompatibility, the ability to cross biological barriers, and the capacity to deliver therapeutic molecules such as miRNAs and proteins. They demonstrate significant potential for enhancing RGC survival and promoting neuroprotection in glaucoma. However, challenges persist, including cargo heterogeneity, incomplete understanding of biodistribution, variable stability and retention in ocular compartments, and the need for scalable production methods.

CONCLUSION: Advancements in EV engineering, focusing on improved targeting, membrane properties, cargo loading, and pharmacokinetics, are essential for their future clinical application. While EV-based therapies should not be viewed as replacements for existing strategies, they represent a promising addition to the neuroprotective landscape for glaucoma and other retinal neurodegenerative conditions. Further research is necessary to overcome translational challenges and facilitate the development of clinically applicable therapies.